US FDA calls for more data on Actelion's Zavesca for Niemann-Pick type C disease

Swiss biotechnology firm Actelion revealed yesterday afternoon that it has received a complete response letter from the US Food and Drug Administration for its supplemental New Drug Application for Zavesca (miglustat) requesting additional preclinical and clinical information. The sNDA had applied for approval of the drug ' which is already marketed gor Gaucher's disease - for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C (NP-C) disease, a rare, neurodegenerative genetic disorder.

The news had little impact of the firm's stock, which rose a modest 0.5% to 47.85 Swiss francs last night, in line with the 0.5% rise in the Stoxx Europe 600 healthcare index, as some analysts said the risk of Actelion becoming a takeover target is on the rise. Possible bidders that were mentioned in the past include Swiss drug major Novartis, the USA's Abbott Laboratories and UK giant GlaxoSmithKline which is Actelion's development partner for the developmental insomnia drug almorexant. Moreover, it comes as something of a surprise, given that, just in January, the FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 10 to three in favor of the benefit/risk profile of Zavesca for NP-C.

However, the FDA decision is still seen as another setback for Actelion, which earlier this month saw its shares plunge 16.2% to 45.90 Swiss francs, its lowest level for four years, after the company announced the initial results of BUILD-3, a clinical study evaluating the safety and efficacy of its lead product bosentan (sold as Tracleer) in patients suffering from idiopathic pulmonary fibrosis (IPF), failed to meet its primary endpoint (The Pharma Letter March 2).